ReviewAmerican journal of clinical dermatology2020
New and Emerging Targeted Therapies for Vascular Malformations.
Review in American journal of clinical dermatology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 57 papers, 2 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
57 citing papers in PubMed, 2 syntheses or guidelines pooled it, 125 citations in OpenAlex.
- Sclerotherapy vs. surgical excision for lymphatic malformations of the head and neck: a systematic review and meta-analysis.European archives of oto-rhino-laryngology : official journal of the European Federation of Oto-Rhino-Laryngological Societies (EUFOS) : affiliated with the German Society for Oto-Rhino-Laryngology - Head and Neck Surgery · 2024Pooled it
- Interventional therapy of extracranial arteriovenous malformations of the head and neck-A systematic review.PloS one · 2022Pooled it
- Efficacy and safety of sirolimus in the treatment of gastrointestinal angiodysplasias.World journal of gastroenterology · 2025Trial
- Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations.JCI insight · 2023Trial
- Clinical differences in sirolimus treatment with low target levels between children and adults with vascular malformations - A nationwide trial.Clinical and translational science · 2023Trial
- Genetically Guided Pharmacotherapy for Structural Neurovascular Lesions.Neurology. Genetics · 2026Article
- A late appearance case of TEK-related mucocutaneous venous malformation treated by Nd:Yag laser.JAAD case reports · 2026Article
- Calcineurin-NFAT-DSCR1.4 signaling as druggable axis in Gαq-R183Q-driven capillary malformations.Angiogenesis · 2026Article
- Vascular malformations: from genetics to therapeutics.EMBO molecular medicine · 2026Review
- Long-Term TIE2 Inhibition in a TEK-Mutated Venous Malformation: A 3-Year Clinical Experience.Biologics : targets & therapy · 2026Article
- PI3K/AKT/mTOR axis in vascular malformations: from molecular insights to targeted clinical trials.Orphanet journal of rare diseases · 2025Review
- Personalized sirolimus regimen for vascular malformations: a retrospective analysis of VASE cohort.Orphanet journal of rare diseases · 2025Article
- A real-world study of sirolimus in the treatment of pediatric head and neck lymphatic malformations.Journal of vascular surgery. Venous and lymphatic disorders · 2025Article
- Alcohol embolization versus non-invasive treatment for pain relief in peripheral venous malformations: a comparative study.Cardiovascular diagnosis and therapy · 2025Article
- ELMO2 is an essential regulator of carotid artery development.Nature communications · 2025Article
- Recent Advances in Interventional Management of Extracranial Vascular Malformations in Children.Seminars in interventional radiology · 2025Review
- RAS Pathway Mutations and Therapeutics in Vascular Anomalies.Pediatric blood & cancer · 2025Review
- Extracranial arteriovenous malformations: towards etiology-based therapeutic management.The Journal of clinical investigation · 2025Review
- Venous Malformations: Unraveling Latest Mechanisms and Bridging Gaps in Targeted Therapy Development.International journal of biological sciences · 2025Review
- Autologous platelet-rich gel for refractory plantar ulcer following ethanol sclerotherapy of peripheral venous malformation: a Case Report.Frontiers in bioengineering and biotechnology · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Vascular malformations are inborn errors of vascular morphogenesis and consist of localized networks of abnormal blood and/or lymphatic vessels with weak endothelial cell proliferation. They have historically been managed by surgery and sclerotherapy. Extensive insight into the genetic origin and molecular mechanism of development has been accumulated over the last 20 years. Since the discovery of the first somatic mutations in a vascular anomaly 10 years ago, it is now recognized that they are perhaps all caused by inherited or somatic mutations in genes that hyperactivate two major intracellular signaling pathways: the RAS/MAPK/ERK and/or the phosphatidylinositol 3 kinase (PIK3)/protein kinase B/mammalian target of rapamycin (mTOR) pathway. Several targeted molecular inhibitors of these pathways have been developed, mostly for the treatment of cancers that harbor mutations in the same pathways. The mTOR inhibitor sirolimus is the most studied compound for the treatment of venous, lymphatic, and complex malformations. Disease responses of vascular malformations to sirolimus have now been reported in several studies in terms of clinical changes, quality of life, functional and radiological outcomes, and safety. Other targeted treatment strategies, such as the PIK3CA inhibitor alpelisib for PIK3CA-mutated vascular malformations, are also emerging. Repurposing of cancer drugs has become a major focus in this rapidly evolving field.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.